SIMPLICITY: Studying Impacts on Malabsorption With Liprotamase in Cystic Fibrosis (SIMPLICITY)

A Phase 3, Open-Label Study Evaluating the Efficacy and Safety of Liprotamase in Subjects With Cystic Fibrosis-Related Exocrine Pancreatic Insufficiency

Liprotamase consists of 3 soluble, non-porcine digestive enzymes, lipase, protease, and amylase, combined in a fixed ratio. Liprotamase is stable in the stomach and can be formulated without enteric coating for administration either as a capsule or as a dosing solution dissolved in water or juice. The purpose of the present study is to provide efficacy and safety data for a new, soluble formulation of liprotamase, Liprotamase Powder for Oral Solution, in Cystic Fibrosis patients with exocrine pancreatic insufficiency (EPI).

Study Overview

• Status: Completed
• Conditions: Cystic Fibrosis; Exocrine Pancreatic Insufficiency
• Intervention / Treatment: Drug: Liprotamase Powder for Oral Solution

• Study Type: Interventional
• Enrollment (Actual): 15
• Phase: Phase 3

Contacts and Locations

Study Locations

• United States
  • Arkansas
    • Little Rock, Arkansas, United States, 72202
      • Investigator Site 139
  • California
    • Orange, California, United States, 92868
      • Investigator site
  • Colorado
    • Aurora, Colorado, United States, 80045
      • Investigator site 114
  • Florida
    • Altamonte Springs, Florida, United States, 32701
      • Investigator Site 117
    • Hollywood, Florida, United States, 33021
      • Investigator Site 138
    • Miami, Florida, United States, 33136
      • Investigator Site 130
  • Georgia
    • Atlanta, Georgia, United States, 30342
      • Investigator site 110
  • Illinois
    • Glenview, Illinois, United States, 60025
      • Investigator site 109
  • Kansas
    • Wichita, Kansas, United States, 67214
      • Investigator site 105
  • Kentucky
    • Louisville, Kentucky, United States, 40202
      • Investigator Site 122
  • Maine
    • Portland, Maine, United States, 04102
      • Investigator Site 132
  • Michigan
    • Ann Arbor, Michigan, United States, 48109-5212
      • Investigator Site 124
  • Nevada
    • Las Vegas, Nevada, United States, 89107
      • Investigator Site 135
  • North Carolina
    • Durham, North Carolina, United States, 27710
      • Investigator Site 118
  • Oklahoma
    • Oklahoma City, Oklahoma, United States, 73104
      • Investigator Site 101
    • Oklahoma City, Oklahoma, United States, 73112
      • Investigator Site 136
  • Pennsylvania
    • Hershey, Pennsylvania, United States, 17033
      • Investigator site 106
  • Texas
    • Dallas, Texas, United States, 75390-9063
      • Investigator site 111
    • Houston, Texas, United States, 77030
      • Investigator Site 116
  • Virginia
    • Richmond, Virginia, United States, 23298
      • Investigator site 112
  • West Virginia
    • Morgantown, West Virginia, United States, 26506
      • Investigator Site 129

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 4 weeks and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• For Part A: males or females ≥7 years of age
• For Part B: males or females 28 days to <7 years
• Diagnosis of cystic fibrosis based on presentation, genotype and/or sweat chloride
• Low fecal elastase
• Fair-to-good nutritional status

Exclusion Criteria:

• History or diagnosis of fibrosing colonopathy
• Distal intestinal obstruction syndrome in 6 months prior to screening
• Receiving enteral tube feedings
• Chronic diarrheal illness unrelated to pancreatic insufficiency
• Liver abnormalities, or liver or lung transplant, or significant bowel resection

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Part A; Liprotamase Powder for Oral Solution in Subjects aged ≥7 years of age	Drug: Liprotamase Powder for Oral Solution; Oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement therapy; Other Names: Liprotamase
Experimental: Part B; Liprotamase Powder for Oral Solution in Subjects aged 28 days to <7 years	Drug: Liprotamase Powder for Oral Solution; Oral, soluble, non-enterically coated, non-porcine, pancreatic enzyme replacement therapy; Other Names: Liprotamase

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety	Number of subjects reporting 1 or more adverse events	1 week

Collaborators and Investigators

• Sponsor: Anthera Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): June 1, 2016
• Primary Completion (Actual): December 1, 2016
• Study Completion (Actual): March 1, 2017

Study Registration Dates

• First Submitted: April 6, 2016
• First Submitted That Met QC Criteria: April 11, 2016
• First Posted (Estimate): April 12, 2016

Study Record Updates

• Last Update Posted (Actual): May 11, 2018
• Last Update Submitted That Met QC Criteria: April 10, 2018
• Last Verified: April 1, 2018

More Information

Terms related to this study

• Keywords: Cystic Fibrosis; Exocrine Pancreatic Insufficiency; Liprotamase; Pancreatic Enzyme Replacement Therapy (PERT)
• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Respiratory Tract Diseases; Lung Diseases; Infant, Newborn, Diseases; Genetic Diseases, Inborn; Pancreatic Diseases; Fibrosis; Cystic Fibrosis; Exocrine Pancreatic Insufficiency
• Other Study ID Numbers: AN-EPI3332

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO